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Clinical Trials

The goal of clinical trials is to find out whether new medical approaches that are being developed are safe and effective and better than those currently being used. Most drugs or medical procedures that are available for patients today went through clinical trial testing.

To help you understand and share this information, you can request our free booklet that summarizes the detailed information in the following sections.

This phase of research is possible only if patients who have the condition that is being studied participate, so a better name for clinical trials would be “patient-assisted research studies.” Clinical trials are an important option for people thinking about lung cancer treatments because the newest treatment approaches are being tested in them.

What is a clinical trial?

Clinical trials are my new hope, my new best friend. At first I was terrified of them; now I’m excited to have options. Hope is 'a chance'!

- Linnea Olson, stage IV adenocarcinoma

Clinical trials are research studies that take place in a medical (clinical) setting. These studies test new medical approaches for their safety and efficacyThe ability of an intervention (for example, a drug or surgery) to produce the desired beneficial effect among a group of volunteer participants.

For people with lung cancer, clinical trials test new ways to:

Find and diagnose lung cancer

Treat lung cancer

Manage symptoms of the lung cancer or side effects of treatment

Prevent lung cancer in the first place (through chemopreventionThe use of drugs, vitamins, or other agents to try to reduce the risk of, or delay the development or recurrence of, cancer, for example)

New lung cancer treatments are always tested in volunteers with the type and stageThe extent of a cancer in the body of lung cancer for which the new treatment is intended. Clinical trials are vital for adding to medical knowledge that can improve the care given to patients. The results of these research studies determine whether new treatments are approved by the FDA for prescribing by doctors for patients outside of clinical trials.

Each clinical trial has its own protocolA detailed plan of a scientific or medical experiment, treatment, or procedure, or study plan. The protocol outlines every aspect of how the clinical trial is to be conducted, including:1,2

The reason for doing the trial

Who can join the trial (called “eligibility requirements” or “inclusion criteria” and “exclusion criteria”)

How long the study will last

What treatment is given, how the treatment is given, and how often the treatment is given (for studies of new treatments)

What medical tests will be done to measure whether the treatment is working

What types of information will be collected about the people taking part

Note: Patients who participate in treatment-focused clinical trials receive either a new treatment/combination of treatments or the treatment that is currently considered the “gold standard”—the best currently available proven treatment. A patient participating in a clinical trial will never be given less than the standard of care.3

Where do clinical trials take place?

There is no one standard for where clinical trials take place. Some are available in just a few places; others have sites at many locations across the United States and in numerous other countries. They take place in locations such as doctors’ offices, community hospitals and clinics, medical centers, cancer centers (including NCI-designated cancer centers), veterans’ and military hospitals, and the National Institutes of Health Clinical Center.4

When you talk with your doctor about treatment options, ask about clinical trials. There may be one that is right for you taking place at your doctor’s office or nearby. You can find out more about whether there is a clinical trial that is right for you further down on this page, in the “Finding a clinical research study that might be right for you” section.

What are the phases of a clinical trial?

Clinical trials to test new cancer treatments involve a series of steps, called phases. There are three main phases of clinical trials. If a new treatment is successful in one phase, it will move on to further testing in the next phase. A drug or device usually has to show success in the first three phases before it is approved by the FDA for broader use.

However, the FDA also has a number of programs to support faster development and review of new drugs for areas of unmet medical need, such as lung cancer. These programs include breakthrough therapy designation, fast track designation, accelerated approval, and priority review. When a treatment is developed through one of those programs, a drug may be approved after strong phase 2 success, for example, and then monitored closely afterward.5

You may also hear the term “phase 4”—this is also called post-marketing surveillance. The goal is to monitor the long-term effectiveness and side effects of an approved treatment after it is on the market with FDA approval.6

Before a drug is studied in humans, it has been studied in the pre-clinical research phase. During that time, the treatment is generally studied in laboratory animals for safety and efficacy.

The following table, taken primarily from an NCI outline of clinical trial phases, shows the numbers of patients who take part and the purpose of the most common phases of a clinical trial.6,7,8

Phase

Goals

Number of Patients

Phase 1

To find a safe dose of a new treatment

To decide how the new treatment should be given (by mouth, in a vein, etc.)

To see how the new treatment affects the human body (whether it causes side effects)

15-30 people

May be a mix of people with different cancers

Phase 2

To find out if the new treatment has an effect on a certain type of cancer, like lung cancer

To see whether the new treatment causes any side effects

Fewer than 100 people

All the same kind of cancer

Phase 3

To compare the efficacy of the new treatment (or new use of a treatment) with the current standard treatment

From 100 to several thousand

All the same kind of cancer

Phase 4

To monitor the long-term effectiveness and side effects after a new treatment has been approved by the FDA

Patients who have been taking the approved drug

Some researchers design trials that combine two phases (phase 1/2 or phase 2/3 trials) in a single protocol. In this combined design, there is a seamless transition between trial phases, which may allow research questions to be answered more quickly or with fewer patients.

How are people assigned to different treatments in clinical trials?

In some phase 2 clinical trials and all phase 3 clinical trials, there are at least two groups to which patients are assigned. Each of these groups receives a different treatment.

The assignments are made randomly, usually by a computer, so that neither the researchers nor the patient knows which group the patient is in. This means that the results are affected as little as possible by factors other than the treatment received by the patient.

A group that includes patients who receive the new treatment is called the investigational groupIn a treatment clinical trial, the group that receives the new treatment being studied. A group that includes patients who receive the current standard therapy is called the control groupIn a clinical trial, the group that does not receive the new treatment being studied.9

When are placebos used in clinical trials?

In lung cancer clinical trials, patients are never given a placebo instead of an effective standard treatment.

Placebos are sometimes used in cancer treatment clinical trials. They are used when a clinical trial is comparing standard treatment plus a new treatment with standard treatment plus a placebo, as in the graphic below. The placebo is designed to look like the medicine being tested, but it is not an active drug. Using a placebo in this way prevents patients and their doctors from figuring out to which treatment group the patients were assigned.

What happens after the clinical trial is over?

After a clinical trial is over, the research team analylzes the results to determine what the next steps should be, based on the efficacy and safety data. A clinical trial may be stopped or may move on to the next phase. A treatment with strong, positive phase 3 results may move on to approval by the US Food and Drug Administration (FDA). Often, results of a clinical trial are published. Check with your research team.8

The clinical trial’s research team may remain in contact with you and let you know about the trial’s findings and conclusions. They may ask you to continue to provide information about your health either through surveys or actual health examinations. This is in addition to the regular care provided by your own doctor.

Who can participate in a clinical trial?

Each clinical trial defines who is eligible to take part. Each trial must include only people who fit the patient traits for that study (the eligibility criteria). Criteria for who can take part in a trial might include:8,10

A specific age range

Gender

The type of lung cancer—could be by histologyThe study of tissues and cells under a microscope and/or molecular mutationAny change in the gene sequence of a cell

The stage of lung cancer

Site of metastasesThe spread of cancer from the primary site, or place where it started, to other places in the body—for example, whether someone has brain (or central nervous system) metastases

Previous treatment history—type of treatment or how many different types of treatment

Medical history

Current health status

Criteria such as these help reduce the medical differences among people in the trial. When people taking part in a trial are alike in key ways, researchers can be more certain that the results are due to the treatment being tested and not to other factors.

Also, some people have health problems besides lung cancer that could be made worse by the treatments in a trial. If you are interested in joining a trial, you will receive medical tests to be sure that you are fit for the trial.

The factors that allow someone to participate in a clinical study are called inclusion criteria, and the factors that disqualify someone from participating are called exclusion criteria.

How are patients in clinical trials kept safe?

People who take part in clinical trials are protected in a number of ways. The federal government has specific rules that help ensure that clinical trials are conducted both safely and ethically.11,12,13

Ways to keep people safe include:

Informed consentA process in which patients are given important information, including possible risks and benefits, about a medical procedure or treatment, a clinical trial, or genetic testing to help them decide if they want to be treated, tested, or take part in the trial process: Researchers must provide patients who are thinking about joining a clinical research study with detailed information about the study. This includes enough information about the trial’s purpose and possible risks and benefits to allow a patient to make the best decision about whether to take part. This information must be given in writing and may be discussed with the doctors and nurses in the research team. If a person decides to take part in a study, he or she will be given an informed consent document to sign, confirming understanding of this information.

This informed consent document is not a contract. Participants always have the right to leave the clinical trial at any time, even if it is not over yet.

Review of the clinical trial protocol (plan) by an institutional review board (IRB)A group of scientists, doctors, clergy, and patient advocates that reviews and approves the detailed plan for every clinical trial: There is an IRB at every healthcare facility that does clinical research. This board includes doctors, researchers, and community members. The IRB’s role is to ensure that the clinical trial protocol is ethical and that the rights and welfare of the people who take part are being protected.

Ongoing monitoring of the clinical trial: Once the clinical trial is under way, it continues to be monitored by the IRB as well as by the sponsor of the trial, the research team conducting the trial, and, for the phase 3 portion of a clinical trial, a Data and Safety Monitoring Board (DSMB)An impartial group that oversees a clinical trial and reviews the results to see if they are acceptable, which decides whether a trial should be changed or closed. The DSMB is made up of doctors, statisticians, and others who are independent of the people, organizations, and institutions that are sponsoring, organizing, and conducting the clinical trial. DSMB members are experts in clinical research and clinical trials. The FDA also meets with researchers and inspects the clinical trial sites.

How does a clinical trial fit in with a person’s overall health care?

The research plan (protocol) for the clinical trial determines how often a participant receives a drug treatment and what exactly that treatment will be. Most often, a person taking part in a clinical trial will continue to receive the rest of his or her health care from their usual doctors. This is true whether the person is receiving the study treatment there or not. The researchers and usual doctors can work together to ensure that the clinical trial protocol will not conflict with any other drugs or treatments that the person is taking.

What are the benefits and risks associated with a clinical trial?

Like with all treatment options, there are both benefits and risks for people who take part in a clinical trial. Before deciding to take part, you should carefully consider both. If you are thinking about taking part in a clinical trial, ask your doctors and the research team as many questions as you need to until you are satisfied that you understand exactly what is involved. Here are lists from the National Cancer Institute of possible benefits and possible risks:10

Possible benefits:

You will have access to a new treatment that is not otherwise available, reflecting the latest thinking in lung cancer research and treatment

The research team will monitor your health closely, providing excellent care

If the treatment being studied is more effective than the standard treatment, you may be among the first to benefit

Even if you do not directly benefit, the information gathered during the clinical trial will still add to knowledge about lung cancer and can help other people

Possible risks:

The new treatment may be no better than, or even as good as, the standard treatment

New treatments may have side effects that doctors do not expect or that are worse than those of the standard treatment

You may be required to make more visits to the doctor or have more hospitalizations than if you were receiving standard treatment

You may need extra tests, and some of these may be uncomfortable or time-consuming

Even if a new treatment has benefits in some patients, it may not benefit you

Health insurance may not cover all patient care costs in a trial. In addition, you may have extra expenses related to extra doctor visits, such as travel and childcare costs

You may have to travel to the place the research treatment is being given

Paying for clinical trials

There are two types of costs that are associated with a clinical trial: patient care costs and research costs. The NCI offers lists of what is included in these costs.14

Patient care costs are costs related to treating your cancer, whether you are in a clinical trial or are receiving standard care. These costs are covered by most health insurance plans, but it is important for you to check to determine exactly what is covered.

Costs include:

Doctor visits

Hospital stays

Lab tests

X-rays and other imaging tests

Standard cancer treatment

Treatment for symptom and side-effect relief

Research costs are costs related to taking part in a clinical trial. These costs are often not covered by health insurance plans, but they may be covered by the trial’s sponsor.* Again, it is important for you to check to determine exactly what is covered.

Costs include include:

The treatment under study

Lab tests performed solely for research purposes

Additional X-rays and imaging tests performed solely for research purposes

Additionally, when you take part in a trial, you may have extra doctor visits that you would not have with standard treatment. These extra visits can add costs for transportation and child care. Information about resources that can provide help with these and other costs can be found in the Support & Survivorship section.

*Clinical trials may be sponsored—developed and supported by—a number of different organizations and entities, including:

Pharmaceutical or biotechnology companies

Academic medical centers

Community hospitals

Government agencies, such as the National Institutes of Health and the US Department of Defense

Physicians

Patient advocacy groups

Finding a clinical trial that might be right for you

If you are considering participating in a clinical trial, start by asking your doctor whether there is one that might be a good match for you in your geographic area.

In addition, here are several resources to help you find one that may be a good match:

LUNGevity offers its own Clinical Trial Finder at https://clinicaltrials.lungevity.org. You can find available clinical trials by type of lung cancer and geographic location. You will also find links to the medical centers at which these clinical trials are taking place. This tool was developed in partnership with Lilly Oncology and uses information from clinicaltrials.gov.

LUNGevity partners with EmergingMed, a clinical trials matching service, to help you with the decision of whether to participate in a clinical trial. EmergingMed helps you identify which lung cancer clinical trials you may be eligible for. The clinical trial navigators can also guide you through the process of getting enrolled if you choose to take part in a clinical trial. Clinical trial navigators are available Monday through Friday from 9:00 am to 5:00 pm ET at 877-769-4834. Learn more about this free service and even fill out an online profile at https://app.emergingmed.com/lcctal/home.

Information about available clinical trials is also available through these websites:

My Cancer Genome:http://www.mycancergenome.org/. This resource is managed by a team of doctors at Vanderbilt University. My Cancer Genome gives up-to-date information on what mutations make cancers grow and related treatment options, including available clinical trials.

Lung Cancer Mutation Consortium (LCMC): http://www.golcmc.com/. Composed of 16 leading cancer centers across the country, LCMC’s goal is to prospectively examine the tumors of patients with the type of advanced (stage IIIB or IV) non-small cell lung cancerA group of lung cancers that are named for the kinds of cells found in the cancer and how the cells look under a microscope called adenocarcinomaOne type of non-small cell lung cancer that usually develops in the cells lining the lungs, and match patients to the best possible therapies, including those being investigated in clinical trials.

Lung Cancer Master Protocol (Lung-MAP): http://www.lung-map.org/. For patients with squamous cell lung cancer, Lung-MAP is a collaboration of many research sites across the country, using a unique approach to matching patients to one of several drugs being developed.

In addition, if you are interested in a specific drug or other treatment that is being developed, you can often find information about studies for that drug on the website of the company developing it.

Which mutations identified in lung cancer are currently being studied in clinical trials?

Currently clinical trials are open for many drugs that inhibit the effect of genomic mutations seen in NSCLC. The targeted treatments are being studied alone and in combination with other targeted agents, chemotherapy, and radiation therapy.

See "Which mutations identified in lung cancer are currently being studied in clinical trials?" in the Targeted Therapy section for information on specific mutations.

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